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Trial · Interventional

CompletedPhase 3Results postedRegistry data

A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

NCTNCT04578496 (opens ClinicalTrials.gov in a new tab)Other idCUV037
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 3
Enrollment
16 participants (actual)
Sponsor
Clinuvel Pharmaceuticals Limited · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
Not stated
Start
11 Jul 2011
Primary completion
27 Feb 2014
Completion
27 Feb 2014
First posted
8 Oct 2020
Last update posted
19 Nov 2020
Conditions
Erythropoietic Protoporphyria

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20112012201320142015
  1. Ph 3

    A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

    NCT04578496

    11 Jul 2011 to 27 Feb 2014

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP patients.

Interventions

  • AfamelanotideDrug

Arms1

  • Experimental

    Afamelanotide

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).

    Time frame · Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

    Registry description

    The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life.

  2. Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)

    Time frame · Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

    Registry description

    The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMedian· Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).

GroupValue (EPP-QoL score)
Afamelanotide27.8
Primary outcomeMedian· Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)

GroupValue (DLQI score)
Afamelanotide-5

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Afamelanotide–0 / 161 / 16

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years up to 75 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * male or female subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies; * aged 18-75 years (inclusive); * provide written informed patient consent prior to the performance of any study-specific procedure. Exclusion Criteria: * any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant; * any allergy to lignocaine or other local anaesthetic to be used during the administration of the study medication; * EPP patients with significant hepatic involvement; * personal history of melanoma or dysplastic nevus syndrome; * current Bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions; * any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations; * acute history of drug or alcohol abuse (in the last 12 months); * female who is pregnant (confirmed by positive serum β-Human Chorionic Gonadotropin (HCG) pregnancy test prior to baseline) or lactating; * females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, intrauterine device); * sexually active men with partners of child-bearing potential not using barrier contraception during the trial and for a period of three months thereafter; * participation in a clinical trial for an investigational agent within 30 days prior to the screening visit; * prior and concomitant therapy with medications which may interfere with the objectives of the study.

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